Analyst Conference Summary

biotechnology

conference date: April 29, 2026 @ 5:30 AM Pacific Time
for quarter ending: March 31, 2026 (Q1, first quarter 2026)

Forward-looking statements

Overview:

Basic data (GAAP):

Revenue was $ million, up % sequentially from $203 million, and down % from $ million year-earlier.

Net income was negative $ million, down sequentially from negative $229 million, and up from negative $ million year-earlier.

EPS (diluted) was negative $, down sequentially from negative $1.41, and down from negative $ year-earlier,

Guidance:

Conference Highlights:

CEO Brett Monia said ""

In Tryngolza for SHTG (severe hypertriglyceridemia) the Phase 3 CORE data, and from 2 other Phase 3 trials, were positive. Showed pancreatitis reduction. Ionis submitted the sNDA in Q4 2025 with PDUFA 6/30/2026. Estimated U.S. sHTG patients over 1 million. Will adjust price for this much larger population. Already has full field team in place in Q1 2026.

Zilganersen for Alexander disease Phase 3 study reported positive results in Q3 2025. NDA submitted in January 2026. PDUFA 9/22/2026. Could launch in 2026.

Donidalorsen (now Dawnzera) for hereditary angioedema (HAE) was approved by the FDA in August 2025. European approval in January 2026. HAE has over 7,000 patients in the US. Independent commercial launch is off to a good start. Believes peak sales could be $500 million per year. Sales ramp will mainly be in 2026.

Spinraza (nusinersen) for SMA higher dose approved by FDA in Q1 2026. 2026 after receiving a CRL earlier in 2025. Approved in Q4 in EU. Salanersen for SMA had postive Phase 1 results, Biogen will take into registrational studies. Salanersen royalty rates would be higher than those for Spinraza, and of course would extend the patent coverage period.

In Q4 2025 Bepirovirsen for chronic hepatitis B achieved its primary endpoint and demonstrated a statistically significant and clinically meaningful functional cure rate in 2 Phase 3 studies. Global filings began in Q1 2026.

In Q4 2025 Sapablursen for polycythemia vera (PV) had positive Phase 2 results, presented at American Society of Hematology (ASH) conference. Ono is advancing sapablursen into Phase 3 development.

Possible Phase 3 readouts in 2026 include Pelacarsen for Lp(a), Eplantersen for ATTR-CM, Ulefnersen for FUS-ALS, and Sefaxerson for IgAN.

Non-GAAP numbers: net income negative $ million, sequentially down from negative $186 million, and down from negative $ million year-earlier. No non-GAAP EPS given.

Cash ended at $ billion, up sequentially from $2.7 billion.

Ionis has a pipeline of about 45 potential drugs, with 12 in clinical development, and 8 in Phase 3 trials. A growing number are wholly-owned.

GAAP Operating expense was $ million, consisting of $ million for cost of goods sold; $ million for R&D and $ million for selling, general and administrative. Operating income was negative $ million. Other income net was negative $ million. Income tax $ million.

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